Predictors of orphan drug approval in the European Union

European Journal of Clinical Pharmacology

Volumn 64, Issue 5, 2008, Pages 545-552

  Heemstra, Harald E ^a   De Vrueh, Remco L ^b   Van Weely, Sonja ^b   Büller, Hans A ^c   Leufkens, Hubert G M ^a  

^a UTRECHT UNIVERSITY   (Netherlands)

^b Steering Committee on Orphan Drugs   (Netherlands)

^c ERASMUS MEDICAL CENTER   (Netherlands)

Author keywords

Drug development; Orphan drugs; Rare diseases; Regulatory affairs

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; ANAGRELIDE; ARSENIC TRIOXIDE; BOSENTAN; BUSULFAN; CARGLUMIC ACID; CELECOXIB; CLADRIBINE; CLOFARABINE; DASATINIB; DEFERASIROX; GALSULFASE; IBUPROFEN; ILOPROST; IMATINIB; MIGLUSTAT; MITOTANE; NITISINONE; OMEGA CONOTOXIN MVIIA; ORPHAN DRUG; OXYBATE SODIUM; PHOTOFRIN; RAZOXANE; SILDENAFIL; SITAXSENTAN; SORAFENIB; SUNITINIB; UNINDEXED DRUG; ZINC ACETATE;

ARTICLE; BIOTECHNOLOGY; DRUG APPROVAL; DRUG CLASSIFICATION; DRUG INFORMATION; DRUG LEGISLATION; DRUG MANUFACTURE; DRUG MARKETING; DRUG RESEARCH; EUROPEAN UNION; PRIORITY JOURNAL; RARE DISEASE; STATISTICAL PARAMETERS; TREATMENT INDICATION;

DRUG APPROVAL; DRUG INDUSTRY; EUROPEAN UNION; HUMANS; ORPHAN DRUG PRODUCTION; PREVALENCE; RARE DISEASES;

EID: 41049085160     PISSN: 00316970     EISSN: None     Source Type: Journal    
DOI: 10.1007/s00228-007-0454-6     Document Type: Article

References (21)

1. Stolk P, Willemen MJC, Leufkens HGM (2006) Rare essentials: drugs for rare diseases as essential medicines. Bull World Health Organ 84:745-751
2. van Weely S, Leufkens HGM (2004) Background paper: orphan diseases. In: Kaplan W, Laing R (eds) Priority medicines for Europe and the world - a public health approach to innovation. World Health Organisation, Geneva
3. Haffner ME, Whitley J, Moses M (2002) Two decades of orphan product development. Nat Rev Drug Discov 1:821-825
4. Haffner ME (2006) Adopting orphan drugs-two dozen years of treating rare diseases. N Engl J Med 354:445-447
5. Wästfelt M, Fadeel B, Henter JI (2006) A journey of hope: lessons learned from studies on rare diseases and orphan drugs. J Intern Med 260:1-10
6. Rinaldi A (2005) Adopting an orphan. EMBO Rep 6:507-510
7. EURORDIS (2005) Rare diseases: understanding this public health priority. http://www.eurordis.org/article.php3?id_article=1296 Accessed 25 Jul 2007
8. US FDA (1983) The Orphan Drug Act, Public Law, 97-141. http://www.fda.gov/orphan/oda.htm. Accessed 12 Jul 2007
9. Benzi G, Ceci A (1997) Drugs trying to get the parents: there will be incentives for the European scientific community to develop research in the field of the orphan drugs. Pharmacol Res 35:89-93
10. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on Orphan Medicinal Products. Official Journal of The European Communities 2000
11. Minghetti PM, Giudici EM, Montanari L (2000) A proposal to improve the supply of orphan drugs. Pharmacol Res 42:33-37
12. Dear JW, Lilitkarntakul P, Webb DJ (2006) Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products. Br J Clin Pharmacol 62:264-271
13. European Commission (2006) Commission Staff Working Document on the experience acquired as a result of the application of Regulation (EC) No 141/2000 on orphan medicinal products and account of the public health benefits obtained. http://ec.europa.eu/enterprise/pharmaceuticals/orphanmp/doc/ orphan_en_06-2006.pdf. Accessed 25 Jul 2007
14. Joppi R, Bertele V, Garattini S (2006) Orphan drug development is progressing too slowly. Br J Clin Pharmacol 61:355-360
15. EMEA (2005) COMP report to the commission in relation to article 10 of regulation 141/2000 on orphan medicinal products. http://www.emea.europa.eu/ pdfs/human/comp/3521805en.pdf. Accessed 25 Jul 2007
16. EMEA (2005) SME Office. http://www.emea.europa.eu/SME/SMEbackground.htm. Accessed 25 Jul 2007
17. Capdeville R, Buchdunger E, Zimmermann J, Matter A (2002) Glivec (STI571, imatinib), a rationally developed, targeted anticancer drug. Nat Rev Drug Discov 1:493-502
18. Haffner ME, Maher PD (2006) The impact of the Orphan Drug Act on drug discovery. Expert Opin Drug Discov 1:521-524
19. Anonymous (2007) Drugs for rare diseases: mixed assessment in Europe (2007) Prescrire Int 16:36-42
20. European Commission (2006) Inventory of community and member states' incentive measures to aid the research, marketing and development and availability of orphan medicinal products. Revision 2005. http://ec.europa.eu/ enterprise/pharmaceuticals/orphanmp/doc/inventory_2006_08.pdf. Accessed 25 Jul 2007
21. European Commission (2007) Register of designated orphan medicinal products. http://ec.europa.eu/enterprise/pharmaceuticals/register/orphreg.htm. Accessed 25 Jul 2007